RNA and oligonucleotide therapies are in the vanguard of scientific research making the transition from the bench to the clinic. These approaches will in time lead to better and more targeted therapies against major neurological diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, amyotrophic lateral sclerosis, frontotemporal dementia, familial dysautonomia and myotonic dystrophy. Many of these technologies are of relevance to other diseases, and we have appended a list of other institutes that may be interested in providing partial support. Tremendous value is gained by bringing together groups and individuals who are working with different technologies in a variety of models of human disease. Few academic meetings cover the focused ground of this meeting, with a specific emphasis on RNA and oligonucleotide therapies. The meeting will bring together cutting-edge basic, translational, and clinical research, and will attract specialists from industr as well as from academia, ensuring a real-world perspective on these important challenges. The narrow emphasis is a strong plus of the Cold Spring Harbor style meeting, driven as much by the openly submitted abstracts as by the invited speakers. The proposed program will include keynote speakers, invited speakers and short talks selected from openly submitted abstracts. Major themes include: Exon Skipping Therapy; siRNA & miRNA ; Aptamers as Therapeutics; Oligonucleotide Therapy; Delivery of RNA Therapeutics; and Long Non-Coding RNAs. Because the program is assembled in part from openly submitted abstracts, the final program is not decided until a couple of months before the meeting, ensuring that the latest advances and discoveries can be included in the meeting. Two of the three organizers are active in the development of novel therapies specifically targeted towards neurological disorders, and so we anticipate a significant proportion of presented science to be relevant to the treatment of neurological and neuromuscular diseases. Participants will be drawn from academic labs, research institutes and biotech/pharmaceutical industries, and will include leaders in the field, established investigators, junior faculty, postdoctoral fellows, graduate students and corporate scientists. Special effort will be made to encourage participation by underrepresented constituencies and next generation scientists in the program. Each session will be chaired by a leading scientist in the field selected by the organizers. Of special importance are the poster sessions, where many participants can present their work in an atmosphere conducive to informal discussion. The meeting will be of moderate size and we expect about 150-200 people to attend, the vast majority of whom will be presenting a poster or talk.